Therapeutic Prospects for Friedreich's Ataxia

Research output: Contribution to journalShort surveypeer-review

19 Scopus citations

Abstract

Friedreich's ataxia (FRDA) is a progressive disease affecting multiple organs that is caused by systemic insufficiency of the mitochondrial protein frataxin. Current therapeutic strategies aim to elevate frataxin levels and/or alleviate the consequences of frataxin deficiency. Recent significant advances in the FRDA therapeutic pipeline are bringing patients closer to a cure.

Original languageEnglish (US)
Pages (from-to)229-233
Number of pages5
JournalTrends in Pharmacological Sciences
Volume40
Issue number4
DOIs
StatePublished - Apr 2019
Externally publishedYes

Keywords

  • frataxin
  • Friedreich's ataxia
  • gene therapy
  • mitochondria
  • oligonucleotide
  • oxidative stress

ASJC Scopus subject areas

  • Toxicology
  • Pharmacology

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